Welcome to Gene Therapy Net

Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials.

FDA experts offer a unanimous endorsement for pioneering gene therapy for blindness

Posted on: 13 October 2017, source: ScienceMag
A pioneering AAV gene therapy from Spark Therapeutics took a giant stride toward an FDA approval yesterday as an outside panel of experts offered their support for getting this game-changing treatment into the market after looking over the data and hearing from some of the severely sight-impaired patients whose lives had been transformed by this therapy. The vote was 16 to 0 favoring the benefit-risk profile of the drug, backing an OK for voretigene neparvovec by the agency’s Cellular, Tissue and Gene Therapies Advisory Committee and providing a compelling reason for the U.S. Food and Drug Administration to follow through with an historic first U.S. approval of a vector-delivered gene therapy.

DNA surgery on embryos removes disease

Posted on: 5 October 2017, source: BBC News
Precise "chemical surgery" has been performed on human embryos to remove disease in a world first, Chinese researchers have told the BBC. The team at Sun Yat-sen University used a technique called base editing to correct a single error out of the three billion "letters" of our genetic code. They altered lab-made embryos to remove the disease beta-thalassemia. The embryos were not implanted. The team says the approach may one day treat a range of inherited diseases.

Gene therapy reverses multiple sclerosis in mice

Posted on: 25 September 2017, source: BioNews
A novel gene therapy can reverse the symptoms and progression of disease in a mouse model of multiple sclerosis (MS). The neurodegenerative illness is caused by the body's own immune system degrading and destroying nerve cells. US researchers have used a gene therapy approach in the livers of affected mice to produce more regulatory T-cells and reduce this autoimmune response. 'Using a clinically tested gene therapy platform, we are able to induce very specific regulatory cells that target the self-reactive cells that are responsible for causing multiple sclerosis,' said Dr Brad Hoffman at the University of Florida, College of Medicine in Gainesville, who led the study. 'Most current therapies for autoimmune diseases such as multiple sclerosis are based on general immune suppression, which has various side effects or complications.'

Gene therapy for cancer halted after patient death

Posted on: 13 September 2017, source: BioNews
Two trials assessing gene therapy for blood cancer have been put on hold by the US Food and Drug Administration (FDA), following a patient fatality. The therapy, known as 'off-the-shelf' CAR-T immunotherapy, used genetically modified immune system T cells to target cancer, yet unlike other trials the cells were taken from a healthy donor instead of the patient. It was hoped this therapy would prove easier and less expensive to produce.

FDA makes the first gene therapy Kymriah available for treatment of pediatric ALL

Posted on: 31 August 2017, source: News Medical Life Sciences
The U.S. Food and Drug Administration (FDA) has issued a press release making the first even gene therapy available for treatment of children with a form of acute lymphoblastic leukemia (ALL). The drug Kymriah (tisagenlecleucel) is the first of its kind that uses gene therapy to treat patients with this condition. The release was updated yesterday identifying the designantions that were granted to Kymriah.

Gene Therapy Corrects Factor VIII Levels in Hemophilia A

Posted on: 7 August 2017, source: Medscape
Patients with hemophilia A who received a gene for coagulation factor VIII (FVIII) packaged in a viral vector had sustained levels of FVIII during 1 year of observation, eliminating spontaneous bleeds[2]. Most of them did not need FVIII infusions even in the case of major trauma or surgery. In a late-breaking abstract session here at the International Society on Thrombosis and Hemostasis 2017 Congress, Dr John Pasi (Barts and the London School of Medicine and Dentistry, Queen Mary University of London, UK) said six out of the seven patients in one dose cohort have achieved normal factor VIII levels.

First gene therapy — 'a true living drug' — on the cusp of FDA approval

Posted on: 14 July 2017, source: Chicago Tribune
Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients' revved-up immune cells to fight the disease, concluding that the therapy's benefits for desperately ill children far outweigh its potentially dangerous side effects. The unanimous recommendation from the Oncologic Drugs Advisory Committee means the treatment could be approved by the FDA by the end of September, forging a new path in the immunotherapy frontier.